Frontotemporal Dementia, Healthy
Conditions
Brief summary
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
Detailed description
This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.
Interventions
BIOLOGICALAL001
Active dose of AL001
OTHERPlacebo
Saline solution administered as a single infusion as palcebo.
Sponsors
Alector Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
Yes
Inclusion criteria
* BMI 18.0-35.0 kg/m2 * 45-120 kg, inclusive * At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up. * Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit. * In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator. * Willingness and able to comply with the study protocol, in the investigator's judgement.
Exclusion criteria
* Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins. * Positive drug or alcohol at screening and prior to first dose * History of alcohol abuse or substance abuse
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs) | 85 days | Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics (PK) of AL001 | 85 days | Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points |
| Maximum plasma concentration (Cmax) for AL001 | 85 days | Evaluate Cmax for serum and CSF concentration of AL001 at specified time points |
| Area under the curve concentration (AUC) for AL001 | 85 days | Evaluate AUC for serum and CSF concentration of AL001 at specified time points |
Countries
Canada, United Kingdom, United States
Outcome results
None listed